This randomized, double-blind, regimen-controlled, phase II, multicenter study will assess the efficacy and safety of two different vismodegib regimens in patients with multiple basal cell carcinoma. Patients will receive vismodegib 150 mg orally once daily either in an intermittent schedule of 12 weeks vismodegib followed by 8 weeks placebo (Arm A) or as 24 weeks induction followed by an intermittent schedule of 8 weeks placebo followed by 8 weeks vismodegib (Arm B). Anticipated time on study treatment is 72 weeks.

Relative reduction (%) from baseline in the number of clinically evident basal cell carcinomas at Week 73 (after 72 weeks of treatment) [ Time Frame: from baseline to Week 73 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Drop-out rate: Discontinuation of study treatment prior to Week 73 for either adverse events or withdrawal of consent by the patient [ Time Frame: approximately 3.5 years ] [ Designated as safety issue: No ]

Relative reduction (%) from baseline in total size of three target basal cell carcinoma lesions in individual patients at Week 73 [ Time Frame: from baseline to Week 73 ] [ Designated as safety issue: No ]

Proportion of patients with at least 50% reduction in the number of basal cell carcinomas at Week 73 in the two treatment regimens [ Time Frame: approximately 3.5 years ] [ Designated as safety issue: No ]

Number of new basal cell carcinomas at Week 73 in the two treatment groups [ Time Frame: approximately 3.5 years ] [ Designated as safety issue: No ]

Recurrence rate up to Week 125 (52 weeks after study drug discontinuation) [ Time Frame: approximately 3.5 years ] [ Designated as safety issue: No ]

Safety: Incidence of adverse events [ Time Frame: approximately 3.5 years ] [ Designated as safety issue: No ]

Patients with multiple basal cell carcinomas, including patients with Gorlin syndrome, with at least 6 clinically evident basal cell carcinomas at the time of randomization, of which 3 measure 5 mm or more in diameter and are considered target lesions. All other lesions are considered to be non-target lesions

Histopathologic confirmation that at least one of the three target lesions is basal cell carcinoma

Women of childbearing potential must agree to use contraception as defined by protocol during treatment and for at least 7 months after completion of study treatment

Male patients with female partners of childbearing potential must agree to use contraception as defined by protocol during treatment and for 2 months after completion of study treatment

Exclusion Criteria:

Inability or unwillingness to swallow capsules

Pregnant or breastfeeding women

Any metastatic basal cell carcinoma

Locally advanced basal cell carcinoma lesion that is considered to be inoperable or to have medical contraindications to surgery

Recent (.i.e. within the past 28 days prior to randomization) or current participation in another experimental drug study

Known or suspected alcohol abuse

One of the following known rare hereditary conditions: galactose intolerance, primary hypolactasia or glucose-galactose malabsorption

Contacts and Locations

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Please refer to this study by its ClinicalTrials.gov identifier: NCT01815840